Michael Ward

 Michael Ward
Michael Ward
NIH/NINDS
Speaker

Presentations at CRISPR in Drug Discovery 2021

Tue23  Mar04:40pm(20 mins)
Advanced iPSC technologies for neurodegenerative disease research

Session: Functional Genomics
Room: Conference Room
Tue23  Mar05:00pm(20 mins)
Joint Q&A Session

Session: Functional Genomics
Room: Conference Room

Profile of Michael Ward

My group studies inherited age-related neurodegenerative diseases, with a focus on discovering overlapping mechanisms of autosomal-dominant frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). Many of the genes responsible for inherited FTD/ALS are now known, but we understand relatively little about how mutations in these genes cause disease or the functional relationship of these genes to each other. To identify converging mechanisms, we employ a combination of unbiased functional genomic and proteomic techniques, cellular and biochemical studies in human iPSC-derived neurons. I also co-direct a new large-scale genome engineering iPSC project, the iPSC Neurodegenerative Disease Initiative, which is in the process of generating hundreds of iPSC models of neurodegenerative diseases and related phenotypic datasets for the research community. Recent discoveries from our group have demonstrated a new mechanism of axonal RNA transport that is disrupted by mutations of the ALS-associated gene ANXA11, and development of a new forward genetic CRISPRi screening platform for iPSC-derived neurons. Our expectation is that these studies will ultimately reveal central disease mechanisms of FTD/ALS and serve as a foundation for the development of effective disease-modifying therapies.

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